Podcast
Questions and Answers
What are the two main processes involved in the production of proteins from DNA?
What are the two main processes involved in the production of proteins from DNA?
Transcription and translation.
What does siRNA stand for?
What does siRNA stand for?
Small interfering RNA.
How does siRNA prevent protein production?
How does siRNA prevent protein production?
siRNA targets and assists in the destruction of mRNA, preventing the translation of proteins.
What is the role of DICER in the antiviral defense system?
What is the role of DICER in the antiviral defense system?
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What is RISC, and what is its function in the siRNA pathway?
What is RISC, and what is its function in the siRNA pathway?
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What is the final step in the siRNA-mediated antiviral defense system?
What is the final step in the siRNA-mediated antiviral defense system?
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What year did Andrew Fire and Craig Mello receive the Nobel Prize?
What year did Andrew Fire and Craig Mello receive the Nobel Prize?
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What is the key difference between siRNA and mRNA?
What is the key difference between siRNA and mRNA?
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What is the primary function of siRNA in gene silencing?
What is the primary function of siRNA in gene silencing?
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Describe the role of the RISC complex in siRNA-mediated gene silencing.
Describe the role of the RISC complex in siRNA-mediated gene silencing.
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What are two main methods for delivering siRNA into cells?
What are two main methods for delivering siRNA into cells?
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What is the primary benefit of utilizing siRNA technology in gene silencing?
What is the primary benefit of utilizing siRNA technology in gene silencing?
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Identify three key challenges that currently limit the widespread use of siRNA therapeutics.
Identify three key challenges that currently limit the widespread use of siRNA therapeutics.
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Mention two specific therapeutic areas where siRNA technology has shown promise.
Mention two specific therapeutic areas where siRNA technology has shown promise.
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What was the key event that marked the FDA approval of the first siRNA-based therapy?
What was the key event that marked the FDA approval of the first siRNA-based therapy?
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Discuss factors that might explain the initial enthusiasm for siRNA technology, followed by a period of slowed progress.
Discuss factors that might explain the initial enthusiasm for siRNA technology, followed by a period of slowed progress.
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Flashcards
siRNA
siRNA
Small interfering RNA that targets mRNA to inhibit protein production.
Transcription
Transcription
The process of creating mRNA from DNA in cells.
Translation
Translation
The process where mRNA is used to synthesize proteins.
Post-translational modification
Post-translational modification
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DICER
DICER
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RISC complex
RISC complex
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Gene silencing
Gene silencing
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Andrew Fire and Craig Mello
Andrew Fire and Craig Mello
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RISC
RISC
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Gene Targeting
Gene Targeting
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Synthesis of siRNA
Synthesis of siRNA
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Therapeutic Areas for siRNA
Therapeutic Areas for siRNA
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Delivery Challenges
Delivery Challenges
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Study Notes
Small Interfering RNA (siRNA)
- siRNA is a method to prevent protein production by targeting and destroying messenger RNA (mRNA).
- siRNA molecules are short RNA fragments that inhibit protein translation.
- siRNA works by targeting mRNA using a RISC complex, which creates a cut that prevents the translation of the protein.
- siRNA's mechanism is similar to the body's antiviral defense system, using DICER to break down viral RNA into smaller fragments.
siRNA's Mechanism of Action
- siRNA sequence is designed to match the mRNA encoding the protein being targeted.
- siRNA is typically double-stranded RNA, around 20 base pairs long, and commercially synthesized.
- The siRNA is introduced into cells using artificial or viral-based methods.
- The RISC complex (containing Ago2 protein) binds one strand of siRNA.
- The siRNA/RISC complex targets and cuts the specific mRNA.
- The cut mRNA is no longer able to create the protein, leading to gene silencing.
siRNA Applications and Challenges
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siRNA targets various tissues, including eyes, lungs, liver, skin, kidney, and tumours.
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siRNA treatment areas include age-related macular degeneration, asthma, metabolic diseases, dermatological conditions, renal failure, and cancer.
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siRNA technology showed promise in the past. Merck acquired Sirna Therapeutics in 2006 but later sold it to Alnylam at a lower price.
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Novartis sold its RNAi business to Arrowhead at a low cost.
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In 2018, Alnylam's siRNA was approved by the FDA for treating a rare disease.
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Main benefit: Ability to design specific siRNA for each protein due to our knowledge of the human genetic code enabling precise downregulation of drug targets.
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Main challenges: siRNA delivery to target cells, cellular uptake, and siRNA stability.
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Novel methods are being developed to overcome these delivery challenges.
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Description
Test your understanding of small interfering RNA (siRNA) and its role in gene silencing. This quiz covers key concepts, mechanisms, and challenges related to siRNA technology, as well as its therapeutic applications. Perfect for students and professionals interested in molecular biology and genetics.